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of the short-chain fatty acid derivative 2,2-dimethylbutyrate (HQK-1001) in inducing Hb F in 76 patients with SCD.  The… Expand This placebo‐controlled phase II study evaluated the pharmacodynamics, efficacy and safety of 2,2‐dimethylbutyrate (HQK‐1001), a… Expand To the editor:
β-thalassemia syndromes comprise a global health burden. Hemoglobin E (HbE)–β thalassemia represents 60% of… Expand The β-thalassaemia syndromes represent a World Health Organization-designated global health burden (Weatherall et al, 2010… Expand 2,2‐dimethylbutyrate (HQK‐1001), an orally‐bioavailable promoter‐targeted fetal globin gene‐inducing agent, was evaluated in an… Expand β‐thalassaemia intermedia (BTI) syndromes cause haemolytic anaemia, ineffective erythropoiesis, and widespread complications… Expand Therapeutics which reduce the pathology in sickle cell syndromes are needed, particularly noncytotoxic therapeutics. Fetal… Expand Abstract 943 Definitive therapies to reduce the underlying pathology in sickle cell syndromes are still needed, particularly non… Expand Abstract 977 Several classes of HbF inducers, including chemotherapeutic agents, ESAs, and short chain fatty acids (SCFADs) have… Expand Development of non-cytotoxic, orally-active therapeutics, which induce fetal globin production, would be beneficial for treatment… Expand